Clinical trials: Gene Mutations
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
- Trial name
- NCT03333486
- Testing
- Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Roswell Park Cancer Institute
Retinitis Pigmentosa, Usher Syndrome Type 2
Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)
The purpose of this study is to evaluate the efficacy safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease
- Trial name
- NCT05158296
- Testing
- Ultevursen
- Where
- 15 sites in United States, Germany and 2 more
- Volunteers
- 7 people
- Sponsor
- Laboratoires Thea
Attention Deficit Hyperactivity Disorder
PART A: Efficacy and Safety of AEVI-001 in Children and Adolescents With ADHD and With mGluR Mutations
This is PART A of a 2-part, 6-week, double-blind, dose-optimization, parallel-group study in children and adolescents (ages 6-17 years) with ADHD with and without CNVs in specific genes implicated in glutamatergic signaling and…
- Trial name
- NCT03265119
- Testing
- AEVI-001
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Aevi Genomic Medicine, LLC, a Cerecor company
Chronic Myelomonocytic Leukemia (CMML)
Cobimetinib in Newly Diagnosed or HMA-treated CMML Patients With RAS Pathway Mutations
This is an open-label, nonrandomized phase 2 trial to assess the efficacy of cobimetinib in RAS pathway activated CMML. All eligible patients will be treated daily with cobimetinib in 28-day cycles.
- Trial name
- CONCERTO (NCT04409639)
- Testing
- Cobimetinib
- Where
- 3 sites in United States
- Volunteers
- 14 people
- Sponsor
- University of Utah
Amyotrophic Lateral Sclerosis
A Study to Assess the Safety, Tolerability, and Effect on Disease Progression of BIIB105 in Participants With Amyotrophic Lateral Sclerosis (ALS) and Participants With the ALS Ataxin-2 (ATXN2) Genetic Mutation
The ALSpire Study is a clinical trial evaluating the investigational drug BIIB105 in adults living with amyotrophic lateral sclerosis (ALS). The ALSpire Study consists of two parts: Part 1: 6-month placebo-controlled study.
- Trial name
- ALSpire (NCT04494256)
- Testing
- BIIB105
- Where
- 17 sites in United States, Canada and 2 more
- Volunteers
- 99 people
- Sponsor
- Biogen
HER2-ve Metastatic Breast Cancer, Germline BRCA1/2 Mutations
To Study Clinical Effectiveness and Safety of Olaparib Monotherapy in Metastatic Breast Cancer Patients.
This open-label, multi-centre phase IIIb study will assess the effectiveness, benefits and potential harms in the use of olaparib monotherapy treatment for patients with HER2-ve metastatic breast cancer associated with germline…
- Conditions
- HER2-ve Metastatic Breast Cancer, Germline BRCA1/2 Mutations, Somatic BRCA1/2 Mutations
- Trial name
- NCT03286842
- Testing
- Olaparib
- Where
- 123 sites in United States, Bulgaria and 13 more
- Volunteers
- 256 people
- Sponsor
- AstraZeneca
Frontotemporal Dementia
A Phase 3 Study to Evaluate Efficacy and Safety of AL001 in Frontotemporal Dementia (INFRONT-3)
A phase 3 double blind, placebo controlled study evaluating the efficacy and safety of AL001 in participants at risk for or with frontotemporal dementia due to heterozygous mutations in the progranulin gene.
- Trial name
- NCT04374136
- Testing
- Part 1 Blinded - AL001, Part 2 (OLE Treatment) - AL001
- Where
- 44 sites in United States, Argentina and 14 more
- Volunteers
- 119 people
- Sponsor
- Alector Inc.
Advanced Malignant Solid Neoplasm, Glioblastoma
Olaparib in Treating Patients With Advanced Glioma, Cholangiocarcinoma, or Solid Tumors With IDH1 or IDH2 Mutations
This phase II trial studies how well olaparib works in treating patients with glioma, cholangiocarcinoma, or solid tumors with IDH1 or IDH2 mutations that has spread from where it first started (primary site) to other places in…
- Conditions
- Advanced Malignant Solid Neoplasm, Glioblastoma, Recurrent Cholangiocarcinoma, Recurrent Glioma
- Trial name
- NCT03212274
- Testing
- Biopsy Procedure, Biospecimen Collection, Computed Tomography
- Where
- 51 sites in United States
- Volunteers
- 89 people
- Sponsor
- National Cancer Institute (NCI)
Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease
A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises
This Phase 3 study will assess the safety and efficacy of inclacumab, a P-selectin inhibitor, in reducing the frequency of vaso-occlusive crises (VOCs) in approximately 240 adult and adolescent participants (≥ 12 years of age)…
- Conditions
- Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease, Vaso-occlusive Crisis
- Trial name
- NCT04935879
- Testing
- Inclacumab
- Where
- 58 sites in United States, Brazil and 10 more
- Volunteers
- 241 people
- Sponsor
- Pfizer
Non-Small Cell Lung Cancer
Phase II Single Arm Study of AZD9291 to Treat NSCLC Patients in Asia Pacific
A Phase II, Open Label, Single-arm Study to Assess the Safety and Efficacy of AZD9291 in Asia Pacific Patients with Locally Advanced/Metastatic Non-Small Cell Lung Cancer whose Disease has Progressed with Previous Epidermal…
- Trial name
- AURA17 (NCT02442349)
- Testing
- AZD9291
- Where
- 30 sites in Australia, China and 1 more
- Volunteers
- 171 people
- Sponsor
- AstraZeneca
Oncology, MET Gene Amplification
Sym015 (Anti-MET) in Patients With Advanced Solid Tumor Malignancies
This is the first study to test Sym015 in humans. The primary purpose of this study is to see if Sym015 is safe and effective for patients with advanced solid tumor malignancies without available therapeutic options.
- Conditions
- Oncology, MET Gene Amplification, NSCLC, MET Gene Mutation
- Trial name
- NCT02648724
- Testing
- Sym015
- Where
- 27 sites in United States, Denmark and 4 more
- Volunteers
- 57 people
- Sponsor
- Symphogen A/S
Carcinoma, Non-Small-Cell Lung
Real World Data on Gi(l)Otrif® Dose Adjustment
This is a non-interventional, multi-country, multi-site study based on existing data from medical records of patients treated with Gi(l)otrif® as part of the routine treatment according to the approved label.
- Trial name
- NCT02751879
- Where
- 29 sites in United States, Austria and 11 more
- Volunteers
- 228 people
- Sponsor
- Boehringer Ingelheim
Gastrointestinal Malignancies
Lurbinectedin in Patients With Advanced Gastrointestinal Malignancies With DNA Repair Mutations
The purpose of this research is to evaluate the activity and safety of lurbinectedin in adult patients with advanced Gastrointestinal Malignancies with DNA repair mutations.
- Trial name
- NCT05229588
- Testing
- Lurbinectedin 4 MG Injection [Zepzelca]
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- HonorHealth Research Institute
Tumor, Solid, Gastrointestinal Cancer
Trial of Ulixertinib in Combination With Hydroxychloroquine in Patients With Advanced Gastrointestinal (GI) Malignancies
This is an open-label, prospective phase two basket trial assessing the efficacy of ulixertinib in combination with hydroxychloroquine in patients with advanced gastrointestinal malignancies.
- Trial name
- NCT05221320
- Testing
- Ulixertinib, Hydroxychloroquine
- Where
- 9 sites in United States
- Volunteers
- 47 people
- Sponsor
- BioMed Valley Discoveries, Inc
Breast Cancer, Metastatic Breast Cancer
ABT-888 and Temozolomide for Metastatic Breast Cancer and BRCA1/2 Breast Cancer
The purpose of this research study is to find out if the combination of ABT-888 and temozolomide is safe and effective in treating patients with metastatic breast cancer.
- Conditions
- Breast Cancer, Metastatic Breast Cancer, BRCA1 Gene Mutation, brca2 Gene Mutation
- Trial name
- NCT01009788
- Testing
- ABT-888, temozolomide
- Where
- 3 sites in United States
- Volunteers
- 63 people
- Sponsor
- Steven J Isakoff, MD, PhD
Carcinoma, Non-Small-Cell Lung
GIOTRIF in First Line Therapy of Advanced NSCLC With EGFR-mutations
This observational study will investigate the efficacy, safety, tolerability and symptom control of GIOTRIF (Afatinib) in daily routine first-line therapy in patients with locally advanced or metastatic NSCLC harboring…
- Trial name
- NCT02047903
- Testing
- Afatinib
- Where
- Germany
- Volunteers
- 161 people
- Sponsor
- Boehringer Ingelheim
RET-fusion Non Small Cell Lung Cancer, Lung Neoplasm
A Study of Pralsetinib Versus Standard of Care for First-Line Treatment of Advanced Non-Small Cell Lung Cancer (NSCLC)
This is an international, randomized, open-label, Phase 3 study designed to evaluate whether the potent and selective RET inhibitor, pralsetinib, improves outcomes when compared to a platinum chemotherapy-based regimen chosen by…
- Conditions
- RET-fusion Non Small Cell Lung Cancer, Lung Neoplasm, Carcinoma, Non-Small-Cell Lung, Respiratory Tract Neoplasms
- Trial name
- AcceleRET-Lung (NCT04222972)
- Testing
- Pralsetinib, Carboplatin, Cisplatin
- Where
- 74 sites in Argentina, Australia and 20 more
- Volunteers
- 223 people
- Sponsor
- Hoffmann-La Roche
Prostate Cancer
Cabozantinib in Patients With Metastatic Castrate Resistant Prostate Cancer (mCRPC)
The purpose of this study is to determine what effects (good and bad) cabozantinib has in treatment of patients with metastatic castrate resistant prostate cancer (mCRPC).
- Trial name
- NCT04631744
- Testing
- Cabozantinib
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Weill Medical College of Cornell University
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia in Remission
Irradiated Donor Cells Following Stem Cell Transplant in Controlling Cancer in Patients With Hematologic Malignancies
This pilot clinical trial studies the side effects of irradiated donor cells following stem cell transplant in controlling cancer in patients with hematologic malignancies.
- Conditions
- Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia in Remission, Hematopoietic Cell Transplantation Recipient, JAK2 Gene Mutation
- Trial name
- NCT03272633
- Testing
- Allogeneic Hematopoietic Stem Cell Transplantation, Irradiated Allogeneic Cells
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Rutgers, The State University of New Jersey
Fabry Disease
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
The primary objective of this study was to compare the effect of migalastat (123 milligrams [mg] of migalastat [equivalent to 150 mg of migalastat hydrochloride]) (migalastat) versus placebo on kidney globotriaosylceramide (GL-3).
- Trial name
- NCT00925301
- Testing
- migalastat hydrochloride
- Where
- 28 sites in United States, Argentina and 11 more
- Volunteers
- 67 people
- Sponsor
- Amicus Therapeutics